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婴儿偏瘫和脑裂畸形与 COL4A1 基因突变相关:古尔德综合征。

Infantile hemiparesis and porencephaly due to a COL4A1 mutation: Gould syndrome.

机构信息

Rocky Vista University College of Osteopathic Medicine, Engelwood, Colorado, USA

Rocky Vista University College of Osteopathic Medicine, Engelwood, Colorado, USA.

出版信息

BMJ Case Rep. 2024 Feb 14;17(2):e259103. doi: 10.1136/bcr-2023-259103.

Abstract

Gould syndrome is an autosomal dominant syndrome due to a COL4A1 or COL4A2 mutation that is commonly characterised by familial porencephaly, seizures, intracranial haemorrhages, cataracts, nephropathies and more. There have been up to 137 identified patients based on a review of the literature. In this case, we describe a male infant that presents with hemiparesis, developmental delay and gait abnormalities at his well-child check. Referral to neurology and a subsequent MRI demonstrated porencephaly and ocular lens abnormalities. Genetic sequencing uncovered a mutation to the COL4A1 gene, suggesting Gould syndrome. There are no family members with similar phenotypes. Mutations to the COL4A1 and COL4A2 genes result in disruption of collagen found in most basement membranes, resulting in a variety of phenotypes that can make diagnosis difficult. Genetic identification of these patients is critical as these patients require a multidisciplinary approach to care and specific counselling on risk reduction techniques.

摘要

古尔德综合征是一种常染色体显性遗传综合征,由 COL4A1 或 COL4A2 基因突变引起,其特征通常为家族性脑裂畸形、癫痫发作、颅内出血、白内障、肾病等。根据文献回顾,已经有多达 137 例已确诊的患者。在本病例中,我们描述了一名男性婴儿,在其定期健康检查时表现为偏瘫、发育迟缓及步态异常。转至神经科并进行随后的 MRI 检查显示为脑裂畸形和晶状体异常。基因测序发现 COL4A1 基因突变,提示为古尔德综合征。家族中没有具有相似表型的成员。COL4A1 和 COL4A2 基因突变导致大多数基底膜中胶原的破坏,导致多种表型,使诊断变得困难。对这些患者进行基因鉴定至关重要,因为这些患者需要多学科的治疗方法,并就降低风险的技术进行专门咨询。

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